Finally, Celgene wins. Anemia drug luspatercept — partnered with Acceleron — scores in PhIII trial

After Celgene’s many trips and blunders this year, the pharma giant might finally please investors Thursday with cheery news from one of its late-stage blockbuster hopefuls. The company reported its anemia drug luspatercept has met primary and secondary endpoints in a Phase III trial.

Celgene $CELG, which is partnered with Acceleron on the drug, said luspatercept “significantly” reduced the need for blood transfusions in patients with the blood disease myelodysplastic syndrome (MDS).

MDS is a bone marrow disease that can lead to a decreased production of red blood cells. Although the company’s announcement was short on details, it did note the Medalist trial had met both its primary endpoint and two secondary endpoints. Most importantly, luspatercept scored a “highly statistically significant” improvement in the percentage of patients who became independent of red blood cell transfusions for at least eight consecutive weeks compared to a placebo.

After the mongersen failure and the company’s embarrassing Ozanimod RTF, Evercore’s Umer Raffat said luspatercept was a “must win.” Leerink’s Geoffrey Porges has said this product is a “standard bearer for Celgene’s late stage pipeline.”

Touted as a $2 billion drug, it’s important to note this isn’t the only Phase III trial Celgene’s waiting on. Celgene’s Commands study is critical, Raffat said.

Jay Backstrom

“This result from the phase III Medalist trial demonstrates the potential clinical benefit of luspatercept as an erythroid maturation agent for the treatment of chronic anemia in patients with low-to-intermediate risk MDS,” said Jay Backstrom, chief medical officer for Celgene. “Based on these results, we look forward to preparing the dossier for global regulatory submissions and also investigating the clinical potential of luspatercept in ESA-naïve, low-to-intermediate risk MDS patients through the initiation of our Phase III Commands study.”

Based on the study results, Acceleron and Celgene plan to submit luspatercept for approval in the US and Europe in the first half of 2019. Celgene’s stock is up 1.2% in after-hours trading Thursday, and Acceleron $XLRN is up 11%.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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